European Medicines Agency review of ataluren for the treatment of ambulant patients aged 5 years and older with Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene

被引：117

作者：

Haas, Manuel ^{[1
]}

Vlcek, Viktor ^{[1
]}

Balabanov, Pavel ^{[1
]}

Salmonson, Tomas ^{[2
]}

Bakchine, Serge ^{[3
]}

Markey, Greg ^{[4
]}

Weise, Martina ^{[5
]}

Schlosser-Weber, Gabriele ^{[5
]}

Brohmann, Henning ^{[5
]}

Prieto Yerro, Concepcion ^{[6
]}

Rodriguez Mendizabal, Macarena ^{[6
]}

Stoyanova-Beninska, Violeta ^{[7
]}

Hiliege, Hans L. ^{[7
]}

机构：

[1] European Med Agcy, London E14 5EU, England

[2] Med Prod Agcy, Lakemedelsverket, Uppsala, Sweden

[3] CHU Reims, Hop Maison Blanche, Reims, France

[4] Med & Healthcare Prod Regulatory Agcy, London, England

[5] Bundesinst Arzneimittel & Med Prod, Bonn, Germany

[6] Agencia Espanola Medicamentos & Prod Sanitarios, Madrid, Spain

[7] Med Evaluat Board, Utrecht, Netherlands

来源：

NEUROMUSCULAR DISORDERS | 2015年 / 25卷 / 01期

关键词：

6-MINUTE WALK TEST; NATURAL-HISTORY; DISEASE PROGRESSION; END-POINTS; MULTICENTER; TRIAL;

D O I：

10.1016/j.nmd.2014.11.011

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

[No abstract available]

引用

页码：5 / 13

页数：9

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